CAMBRIDGE, Mass., June 17,
2024 /PRNewswire/ -- Kira Pharmaceuticals, a global
biotechnology company pioneering transformational complement
therapies to treat immune-mediated diseases, today announced
positive long-term results from its Phase 2 study of KP104 in
complement inhibitor-naïve patients with PNH. KP104 a
first-in-class dual-targeting complement inhibitor (C5 & Factor
H) of both alternative and terminal pathways. The findings were
presented at the 2024 European Hematology Association (EHA) Hybrid
Congress in Madrid, Spain,
highlighting KP104's transformative potential as a novel
monotherapy for PNH to address the existing unmet medical
needs.
Key highlights from the study include results from 18 patients
treated with KP104 subcutaneously for up to 65 weeks, with 24- 26
weeks of the treatment time being on Optimal Biological Dose (OBD)
for all patients, switched from 3 cohorts (n=6 per cohort) in dose
escalation phase.
- Patients continued to show sustained improvements in
hemoglobin levels after switching to OBD (Figure
1): 100% (18/18) achieved a ≥2 g/dL increase from
baseline, and 89% (16/18) attained hemoglobin normalization (≥12
g/dL) with an average (SD) of 13.5 (1.4) g/dL in the absence of RBC
transfusions. The remaining two patients with co-existing
conditions, one with aplastic anemia (AA) and the other with
myeloproliferative neoplasms (MPN), also demonstrated hemoglobin
improvement approaching near-normal levels.
- Patients showed sustained control of LDH
levels (Figure 2) to near-normal level
throughout the entire treatment period, demonstrating strong IVH
control. At the end of 24/26 weeks post-OBD switch, 94% (17/18)
patients achieved LDH <1.5x ULN.
- All patients remained free from RBC transfusions between day
1 and week 65 of KP104 treatment.
- Significant clinical improvements were also observed in all
other secondary endpoints: normalization of absolute
reticulocyte counts, bilirubin levels, and FACIT-fatigue scores
after switching to OBD.
- KP104 was safe and well-tolerated, and produced no
treatment-emergent adverse events (TEAEs) at or above grade
3.
The affirming long-term results from the Phase 2 study further
demonstrate KP104's potential as an optimal first-line monotherapy
to safely and effectively control both intravascular and
extravascular hemolysis of PNH. Global Phase 3 studies are being
planned to potentially establish KP104 as the new standard of care
for PNH.
"We are very encouraged by the robust efficacy and
favorable safety profile demonstrated by KP104 in our Phase 2
study," said Dr. Wenru Song, Head of
R&D at Kira Pharmaceuticals, "these long-term results
support the advancement KP104 into Phase 3 trials. We are committed
and look forward to bringing this promising therapy to patients
suffering from PNH as quickly as possible."
Oral Presentation Details:
- Title: KP104, a bifunctional C5 mAb-Factor H fusion
protein, effectively controls intravascular and extravascular
hemolysis in complement inhibitor-naïve PNH patients: long-term
results from a phase 2 study
- Authors: Bing Han, Fengkui Zhang, Li Zhang, Chen
Yang, Changhe Yue,
Chunrong Wang, Jay Ma, Chaomei
He, Ping Tsui, Jingtao Wu, Qing Yu Christina Weng, Richard Lee, Helen
Fu, Hui Yan, Wenru Song
- Session: s454 Clinical and Translational in Bone
Marrow Failure and PNH
- Time: June 15, 2024, 16:30 -
17:45 CEST
The presentation slides will be available on Kira's website at
https://www.kirapharma.com/publications.
About KP104
KP104 is a first-in-class bifunctional
biologic designed to simultaneously block both the alternative
(Factor H) and terminal (C5) complement pathways, providing a
powerful and synergistic method of targeting the validated drivers
of complement-mediated disease. This dual-target mechanism of
action uniquely positions KP104 to address complement-mediated
diseases and potentially provide greater benefits than
single-target complement agents. Engineered to have an extended
half-life and enhanced potency, KP104 has a formulation suitable
for both intravenous and subcutaneous administrations. KP104 is
entering Phase 2 POC trials across multiple renal disease and
hematologic indications and has been granted Orphan Drug
Designation by the FDA for the treatment of paroxysmal nocturnal
hemoglobinuria. Phase 2 trials will be conducted globally,
including in the U.S., China, and
Australia. KP104 is an
investigational agent not yet approved for any indication by any
health authority.
About Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal
Nocturnal Hemoglobinuria is a rare, life-threatening blood disease
that is characterized by the destruction of red blood cells,
formation of blood clots, and impairment of bone marrow function.
PNH affects between 1 and 5 people per million and is almost always
caused by a genetic mutation that results in production of aberrant
hematopoietic stem cells. These stem cells produce irregular red
blood cells that are highly susceptible to destruction via
complement activation. Current therapies include C5 inhibitors,
which do not address extravascular hemolysis (EVH) related to the
alternative pathway or a C3 inhibitor, which may address EVH but
may not adequately block C5 downstream, leading to life-threatening
breakthrough hemolysis (Breakthrough Hemolysis in PNH with Proximal
or Terminal Complement Inhibition, N Engl J Med, July 14, 2022).
About Kira Pharmaceuticals
Kira Pharmaceuticals is a
clinical-stage biotechnology company pioneering complement-
targeted therapies to treat immune-mediated diseases. Enabled by
its LOGIC platform, the company has developed a robust pipeline of
novel assets against validated complement targets. Headquartered in
Cambridge, Massachusetts and with
facilities in China and
Australia, Kira Pharmaceuticals
has established a global team committed to advancing life-changing
therapies to patients around the world. More information on Kira
can be found at www.kirapharma.com and on LinkedIn.
CONTACT: Kiramedia@kirapharma.com
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